The AMP BGTC brings together partners from the public, private, and non-profit sectors to foster development of gene therapies intended to treat rare genetic diseases, which affect populations too small for viable commercial development.
ASC Therapeutics is a biopharmaceutical company pioneering the development of gene replacement therapies, in-vivo gene editing and allogeneic cell therapies for hematological, metabolic, and other rare diseases.
The Accelerating Medicines Partnership program is a public-private partnership between the National Institutes of Health, the US Food and Drug Administration, and multiple public and private organizations.
The AMP BGTC aims to develop platforms and standards that will speed the development and delivery of customized or 'bespoke' gene therapies that could treat the ms of people affected by rare diseases.
The FNIH creates and manages alliances with public and private institutions in support of the mission of the NIH. The FNIH works with its partners to accelerate biomedical research and strategies against diseases and health concerns in the United States and across the globe.
Established by Congress in 1990, the FNIH is a not-for-profit 501(c) (3) charitable organization.
CARsgen Therapeutics' satri-cel NDA accepted by Chinese regulator
Sanofi receives FDA orphan drug designation for riliprubart in transplant rejection
Hoth Therapeutics reports positive interim results for HT-001 topical therapy
Breckenridge Pharmaceutical's generic for Ablysinol granted final FDA approval
Hemab Therapeutics presents bleeding disorder clinical and preclinical data at ISTH 2025 Congress
Cumberland Pharmaceuticals reports ifetroban Phase 2 DMD heart disease trial results
Sarclisa recommended for EU approval in newly diagnosed transplant-eligible multiple myeloma